Top Regulatory News Stories – Week Ending August 2, 2019

Celgene received FDA approval for Otezla to treat oral ulcers associated with Behçet’s Disease.

Apic Bio, Inc. announced that the FDA has granted orphan drug designation to APB-102, a gene therapy soon to be in clinical development for the treatment of genetic SOD1 amyotrophic lateral sclerosis (ALS).

Biohaven Pharmaceuticals reported that the FDA failed to approve its treatment for amyotrophic lateral sclerosis, or Lou Gehrig’s disease.

Baxter International announced the FDA has approved Myxredlin as the first and only ready-to-use insulin for IV infusion in the hospital and other acute care settings.

Vertex Pharmaceuticals announced the submission of a NDA to the FDA for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen.

Seelos Therapeutics announced that it has submitted an IND application with the U.S. Food and Drug Administration (FDA) for the initiation of a Phase IIb/III clinical study of SLS-005 (trehalose) for the treatment of Sanfilippo syndrome (SFS).

Myovant is preparing to seek regulatory approval for its treatment of uterine fibroids following positive Phase III results that showed a 71.2% response rate in patients.

Nektar Therapeutics reported that its opioid pain drug NKTR-181 was being put on hold at the FDA.

Orchard Therapeutics announced that it has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for OTL-103, Orchard’s ex vivo autologous hematopoietic stem cell (HSC)-based gene therapy for the treatment of Wiskott-Aldrich Syndrome (WAS)

In other news…

Pfizer plans to combine its Upjohn off-patent branded and generic established medicines business with Mylan into a new company:

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